60 Participants Needed

LUM-201 for Growth Hormone Deficiency

(OraGrowtH211 Trial)

Recruiting at 6 trial locations
LP
Overseen ByLumos Pharma
Age: < 18
Sex: Any
Trial Phase: Phase 2
Sponsor: Lumos Pharma
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial

Trial Summary

What is the purpose of this trial?

This is a multi-national trial. The trial aims to study the long-term safety of LUM-201 in subjects with Idiopathic Pediatric Growth Hormone Deficiency (iPGHD). This study will also assess pharmacodynamics and efficacy response to therapy with LUM-201.

Will I have to stop taking my current medications?

You may need to stop taking certain medications that affect the cytochrome system CYP3A4, which is involved in processing LUM-201. If you're on these medications for more than two weeks, you might not be able to participate, but shorter-term use will be evaluated individually.

What data supports the effectiveness of the drug LUM-201 for treating growth hormone deficiency?

Research shows that LUM-201, a drug that helps the body release more growth hormone, leads to greater growth hormone responses in children with growth hormone deficiency compared to standard treatments.12345

How is the drug LUM-201 different from other treatments for growth hormone deficiency?

LUM-201 is unique because it is an oral medication that stimulates the body's own growth hormone production, unlike traditional treatments that involve injections of synthetic growth hormone.15678

Eligibility Criteria

This trial is for children with Idiopathic Pediatric Growth Hormone Deficiency who have already been part of a previous LUM-201 study. They must have completed at least 12 months in that study and be cleared to continue treatment. Children with conditions that add risk or those on certain long-term medications affecting LUM-201 metabolism cannot participate.

Inclusion Criteria

You were part of a previous study for pediatric LUM-201 GHD treatment and completed at least the 12-month visit. If you meet all other requirements, you may be able to continue the treatment.
Parent/caregiver must sign the informed consent, and the subject must sign the assent, as applicable.

Exclusion Criteria

I am on long-term medication that affects how my body processes certain drugs.
Medical or genetic condition that, in the opinion of the PI and/or MMs, adds unwarranted risk to the use of LUM-201

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Extension

Participants continue LUM-201 treatment to monitor long-term safety and efficacy

36 months
7 in-person visits with follow-up phone calls between visits

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

  • LUM-201
Trial OverviewThe trial is testing the long-term safety of a drug called LUM-201 in children with growth hormone deficiency. It will also look at how the body responds to the drug over time and its effectiveness in promoting growth.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: LUM-201 (1.6 mg/kg/day)Experimental Treatment1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

Lumos Pharma

Lead Sponsor

Trials
5
Recruited
180+

Findings from Research

A systematic review of 94 studies indicates that both short-term and long-term growth hormone (GH) replacement therapy in adults with GH deficiency is generally safe, showing no increased risk of diabetes mellitus or cancer recurrence.
Despite the positive findings, the review highlights limitations such as the small number of subjects and insufficient long-term data, which may affect the reliability of the conclusions drawn.
Reviewing the safety of GH replacement therapy in adults.Stochholm, K., Johannsson, G.[2015]
A study involving 135 adults with adult growth hormone deficiency (AGHD) found that both depot growth hormone (GH) given every 14 days and daily GH injections significantly reduced trunk and visceral fat, with the depot form showing a 3.2% decrease in trunk fat compared to no treatment.
Both treatment methods increased lean body mass and had similar side effect profiles, indicating that the depot GH is as safe and effective as daily injections for managing AGHD.
Efficacy of a long-acting growth hormone (GH) preparation in patients with adult GH deficiency.Hoffman, AR., Biller, BM., Cook, D., et al.[2016]

References

Safety and Efficacy of Lonapegsomatropin in Children With Growth Hormone Deficiency: enliGHten Trial 2-Year Results. [2022]
Reviewing the safety of GH replacement therapy in adults. [2015]
A monocentric experience of growth hormone replacement therapy in adult patients. [2016]
Efficacy of a long-acting growth hormone (GH) preparation in patients with adult GH deficiency. [2016]
A GH Secretagogue Receptor Agonist (LUM-201) Elicits Greater GH Responses than Standard GH Secretagogues in Subjects of a Pediatric GH Deficiency Trial. [2022]
Development of a Predictive Enrichment Marker for the Oral GH Secretagogue LUM-201 in Pediatric Growth Hormone Deficiency. [2022]
The effect of long-term growth hormone (GH) treatment on bone mineral density in children with GH deficiency. Role of GH in the attainment of peak bone mass. [2022]
Childhood onset of GH deficiency: reassessment of GH status and effects of substitution. [2019]